Regulatory Authority Actions

Acoramidis was approved by FDA in November 2024 and is prescribed in the US under the brand name Attruby.

While this approval addresses breast cancer, olaparib in combination with durvalumab was approved by the EU in August 2024 for treatment of endometrial cancer.

These infections can be caused by certain Gram-negative microorganisms, making the infections challenging to control due to high antimicrobial resistance.

EMA introduced its New Fee Regulation in January 2025, but what impact will the new fees and charges have on medicine developers?

Bio/pharma companies have a lot to consider when tackling social media communications, especially within Europe.

Another eight medications already authorized in the EU were recommended for extensions of various indications.

Drug manufacturer Novo Nordisk said the approval establishes Ozempic as the most broadly indicated GLP-1 RA in its class.

Data from a Phase III study that evaluated lazertinib, in combination with amivantamab, in comparison to osimertinib as a first-line treatment resulted in the EC’s decision.

Imfinzi received an expanded indication for lung cancer from FDA in August 2024, and Tagrisso had been approved to treat lung cancer in China in June 2024.

The agency’s newly issued letter to Sanofi amends one sent in December 2024, and stems from inspections at its Massachusetts drug manufacturing facility in June and July 2024.

Patrizia Cavazzoni, director of FDA’s Center for Drug Evaluation and Research, has announced her retirement from the agency.

Posdinemab is the second tau-directed investigational therapy by J&J that has received fast track designation from FDA this year for Alzheimer’s disease.

The final guidance is a revision of a policy that has existed in some form since at least 2009, while a separate, draft guidance will accept public comment for consideration for its final version through April 8.

With this approval, HYQVIA [Immune Globulin Infusion 10% (human) with Recombinant Human Hyaluronidase] becomes the first and only facilitated subcutaneous immunoglobulin available in Japan to treat these disorders.

The comment period for the Federal Register Notice, “Evaluating the Immunogenicity Risk of Host Cell Proteins in Follow-on Recombinant Peptide Products: Establishment of a Public Docket: Request for Information and Comments”, has been reopened by FDA until March 3, 2025.

FDA's approval of Hikma Pharmaceuticals’ liraglutide injection makes this product the first generic version of Victoza, a GLP-1 receptor agonist.

The Center for Drug Evaluation and Research’s new center will coordinate and promote use of real-word data in regulatory decisions.

FDA's approval will allow Kedrion to manufacture Ryplazim (plasminogen, human-tvmh), the only FDA-approved therapy for treating PLGD-1, at facility in Bolognana, Italy.

FDA's approval of eladocagene exuparvovec-tneq (Kebilidi) makes that product the first FDA-approved gene therapy for treating AADC deficiency.

CHMP has granted Eisai Europe and Biogen a positive opinion for the use of their monoclonal antibody therapy in treating early Alzheimer’s disease.

Balversa (erdafitinib) marks the first and only bladder cancer therapy to target FGFR3 alterations, with a demonstrated increase in overall survival from 7.8 months to 12.1 months.

CDER Director Patrizia Cavazzoni and CBER Director Peter Marks provided an update on the rare disease innovation hub in a new FDA Voices blog post.

Vyalev, also known as Produodopa, has been approved in 35 countries, with more than 4200 patients having started treatment.

As many as 30% of people living with severe hemophilia A develop inhibitors such as TFPI, which can be produced by the body’s immune response to clotting factors in replacement therapy, often limiting the effectiveness of that therapy.

Wainzua had been previously approved for use by patients with ATTRv-PN in the United States, under the brand name Wainua.

The new mechanism of action targets cholinergic receptors instead of the standard dopamine receptors.

The agency is recommending that the market authorization for Oxbryta (voxelotor) be suspended so that emerging data may be reviewed.

Bio/pharma companies should be approaching their decarbonization journey as a strategic business move for the future as industry stakeholders are placing greater value on sustainability.

Phase III trial results showed that Imfinzi (durvalumab in combination with chemotherapy reduced the risk of recurrence, progression, or death by 32% in patients with resectable non-small cell lung cancer before and after surgery.

PiaSky (crovalimab) has previously been approved in countries such as the United States and Japan for monthly, subcutaneous use that may be self-administered with adequate training.